Phase 1/1b Study of MGCD516 in Patients With Advanced Cancer

MGCD516 is a receptor tyrosine kinase (RTK) inhibitor shown in preclinical models to inhibit
a closely related spectrum of RTKs including MET, AXL, MER, and members of the VEGFR, PDGFR,
DDR2, TRK and Eph families. In this study, MGCD516 is orally administered to patients with
advanced solid tumor malignancies to evaluate its safety, pharmacokinetic, metabolism,
pharmacodynamic and clinical activity profiles.

During the Phase 1 segment, the dose and regimen of MGCD516 will be assessed; during the
Phase 1b segment, the clinical activity of MGCD516 will be evaluated in selected patient
populations.

Patients anticipated to be enrolled in Phase 1b will be selected based upon having a tumor
type, including but not limited to, non small cell lung cancer and head and neck cancer
positive for specific activating MET, NTRK2, NTRK3, or DDR2 mutations, MET or KIT/PDGFRA/KDR
gene amplification, selected gene rearrangements involving the MET, RET, AXL, NTRK1, or NTRK3
gene loci, or having loss of function mutations in the CBL gene. In addition patients with
clear cell renal cell carcinoma refractory to angiogenesis inhibitors or metastatic prostate
cancer with bone metastasis will be enrolled.